This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA.
Name: rAAV2-ND4
Description: The Best Corrected Visual Acuity
Measure: BCVA Time: Change from Baseline at 12 months .Description: visual evoked potential
Measure: VEP Time: Change from Baseline at 12 months .Description: retinal nerve fiber layer
Measure: RNFL Time: Change from Baseline at 12 months .Allocation: N/A
Single Group Assignment
There is one SNP
A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy This study is meant to evaluate the safety and efficacy of rAAV2-ND4 treatment for Leber hereditary optic neuropathy with the G11778A mutation in mitochondrial DNA. --- G11778A ---
This is an open-label, single-arm, multi-center study to further evaluate the safety and efficacy of rAAV-ND4 in the treatment of LHON patients with G11778A mutation. --- G11778A ---